Assessment of Patient-Reported Outcomes

Frei, Anja; Puhan, Milo

doi:10.1007/978-3-319-65888-9_7

Cited by 1 publication

(2 citation statements)

References 43 publications

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“…Overall, the PROM development framework will provide the rationale regarding what outcomes to measure and how this is to be accomplished. It should also determine the target population and research application, which will, in turn, define the instrument’s characteristics ( 20 , 22 , 23 ).…”

Section: Types Of Patient Experience Data and Collection Methodsmentioning

confidence: 99%

“…If a questionnaire is developed as a PROM to evaluate how one therapeutic causes breathlessness, the content validity of this instrument would be the extent to which it would measure breathlessness, instead of anxiety or shallow breathing, or any other situations that could be mistaken with the actual claim it is intended to measure (22,23).…”

Section: Content Validitymentioning

confidence: 99%

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Leveraging patient experience data to guide medicines development, regulation, access decisions and clinical care in the EU

Almeida,

Umuhire,

Gonzalez-Quevedo

et al. 2024

Front. Med.

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Patient experience data (PED), provided by patients/their carers without interpretation by clinicians, directly capture what matters more to patients on their medical condition, treatment and impact of healthcare. PED can be collected through different methodologies and these need to be robust and validated for its intended use. Medicine regulators are increasingly encouraging stakeholders to generate, collect and submit PED to support both scientific advice in development programs and regulatory decisions on the approval and use of these medicines. This article reviews the existing definitions and types of PED and demonstrate the potential for use in different settings of medicines’ life cycle, focusing on Patient-Reported Outcomes (PRO) and Patient Preferences (PP). Furthermore, it addresses some challenges and opportunities, alluding to important regulatory guidance that has been published, methodological aspects and digitalization, highlighting the lack of guidance as a key hurdle to achieve more systematic inclusion of PED in regulatory submissions. In addition, the article discusses opportunities at European and global level that could be implemented to leverage PED use. New digital tools that allow patients to collect PED in real time could also contribute to these advances, but it is equally important not to overlook the challenges they entail. The numerous and relevant initiatives being developed by various stakeholders in this field, including regulators, show their confidence in PED’s value and create an ideal moment to address challenges and consolidate PED use across medicines’ life cycle.

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Section: Types Of Patient Experience Data and Collection Methodsmentioning

confidence: 99%

Section: Content Validitymentioning

confidence: 99%